GenSight Biologics Confirms Sustained Efficacy and Safety of Bilateral LUMEVOQ Injections Four Years After One-Time Administration - Seite 2

“The sustained effect on vision, as observed in the REFLECT trial, is a crucial piece of the LUMEVOQ story for patients, physicians and health authorities,” commented Laurence Rodriguez, Chief Executive Officer of GenSight Biologics. “The durable impact from a single administration differentiates gene therapy from other treatment modalities, facilitating patient adherence and improving quality of life.”

Table 1: Change in Best-Corrected Visual Acuity (BCVA) versus Nadir 4 Years after Injection

	4-year
	1st affected eye	2nd affected eye
Subjects bilaterally injected with LUMEVOQ	LUMEVOQ -0.40 LogMAR +20 ETDRS letters	LUMEVOQ -0.34 LogMAR +17 ETDRS letters
Subjects unilaterally injected with LUMEVOQ	LUMEVOQ -0.38 LogMAR +19 ETDRS letters	PLACEBO -0.27 LogMAR +14 ETDRS letters
Data cut-off: Feb 20, 2024. Subjects bilaterally treated: 1st affected eyes: n=48; 2nd affected eyes: n=48; subjects unilaterally treated: 1st affected eyes: n=50; 2nd affected eyes: n=50. p<0.0001 for all eye groups using linear mixed model.

Responder analyses reinforce the finding of improved outcomes for patients, for whom natural history typically results in greatly impaired vision with a very low likelihood of spontaneous recovery.³ Four years after a bilateral injection, 73% of patients had experienced a clinically meaningful improvement of at least -0.3 LogMAR (+15 ETDRS letters) relative to their observed nadir. 81% of bilaterally treated patients are able to read letters on a screen (on-chart vision), with the likelihood of reaching this level of vision being twice as high with bilateral treatment versus a unilateral injection (odds ratio: 2.0 [0.7; 5.5]).

Favorable safety profile

The favorable safety profile of LUMEVOQ continued to be confirmed, with the safety profile of the drug being demonstrated as comparable in bilaterally and unilaterally treated subjects. There was no study discontinuation related to systemic or ocular adverse events, and there were no serious ocular adverse events. The main ocular adverse event was intraocular inflammation, which was mostly mild and responsive to conventional treatment.